—If approved by the European Commission (EC),
VYNDAQEL® will be the first pharmacologic therapy in the EU for
patients with transthyretin amyloid cardiomyopathy—
Pfizer Inc. (NYSE: PFE) announced today that the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines
Agency (EMA) has adopted a positive opinion recommending the
approval of VYNDAQEL® (tafamidis), a once-daily 61 mg oral capsule,
for the treatment of wild-type or hereditary transthyretin
amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
“The CHMP positive opinion of VYNDAQEL for ATTR-CM reflects our
steadfast commitment to improving outcomes for patients living with
this rare and fatal disease,” said Brenda Cooperstone, MD, Senior
Vice President and Chief Development Officer, Rare Disease, Pfizer
Global Product Development. “In ATTR-ACT, VYNDAQEL reduced
mortality and the frequency of cardiovascular-related
hospitalizations in patients with wild-type or hereditary forms of
the disease. If approved, VYNDAQEL would represent a real
breakthrough for patients.”
ATTR-CM is a rare, life-threatening disease characterized by the
buildup of abnormal deposits of misfolded protein called amyloid in
the heart and is defined by restrictive cardiomyopathy and
progressive heart failure.1,2,3 On average, patients live only 2 to
3.5 years following diagnosis.4
“For those living with ATTR-CM, a progressive and fatal rare
disease, there are currently no available pharmacologic treatments
for patients,” said Jean-Christophe Fidalgo, President of the
Amyloidosis Alliance. “The Amyloidosis Alliance applauds the CHMP
opinion, and we hope the EC will swiftly approve VYNDAQEL for
ATTR-CM so patients can receive timely access to this
medicine.”
In 2011, a different form of VYNDAQEL, tafamidis meglumine 20 mg
capsule, was approved in the EU for transthyretin amyloidosis in
adult patients with stage 1 symptomatic polyneuropathy (ATTR-PN) to
delay peripheral neurologic impairment. For ATTR-CM, the tafamidis
61 mg capsule corresponds to an 80 mg tafamidis meglumine dose (4x
20mg capsules) and was developed for patient convenience to enable
a single capsule for daily administration.
The European line extension application was based on the Phase 3
ATTR-ACT study, the first and only completed global, double-blind,
randomised, placebo-controlled clinical trial to investigate a
pharmacologic therapy for the treatment of ATTR-CM.5 In the primary
analysis of the study, VYNDAQEL (tafamidis meglumine) demonstrated
a significant reduction in the hierarchical combination of
all-cause mortality and frequency of cardiovascular-related
hospitalisations compared to placebo over a 30-month period in
patients with wild-type or hereditary ATTR-CM (p=0.0006).5
Additionally, individual components of the primary analysis
demonstrated a relative reduction in the risk of all-cause
mortality and frequency of cardiovascular-related hospitalization
of 30% (p=0.026) and 32% (p<0.0001), respectively, with VYNDAQEL
versus placebo.5 The application is also based on findings from an
evaluation of the free acid form of tafamidis 61 mg.6 The ATTR-ACT
primary results were presented in a Hot Line session at the ESC
Congress 2018 in Munich, Germany, and simultaneously published
online in the New England Journal of Medicine (NEJM) in August
2018.
The CHMP’s opinion will now be reviewed by the EC and a final
decision is expected in the coming months.
About ATTR Amyloidosis
ATTR amyloidosis is rare, progressive disease characterized by
the abnormal buildup of amyloid deposits composed of misfolded
transthyretin protein in the body’s organs and tissues. ATTR
amyloidosis can impact numerous organs and tissues in the body,
including the peripheral nervous system, and organs such as the
heart, kidneys, gastrointestinal tract, and eyes. ATTR-CM and
ATTR-PN are two presentations of the disease.
ATTR-CM affects the heart and leads to restrictive
cardiomyopathy and progressive heart failure. There are two
sub-types of ATTR-CM: hereditary, which is caused by a mutation in
the transthyretin gene and can occur in people as early as their
50s and 60s; or the wild-type form which is associated with aging,
and is thought to be more common, usually affecting men after age
60.7,8 Often ATTR-CM is diagnosed only after symptoms have become
severe. Once diagnosed, the median life expectancy in patients with
ATTR-CM, dependent on sub-type, is approximately two to 3.5
years.4
ATTR-PN results from a genetic mutation of the transthyretin
gene, amyloid fibrils form in the peripheral and autonomic nerves.
ATTR-PN typically occurs during active adult years with onset as
early as the 30s in some patients, followed by disease progression
that may reach the terminal stage in approximately 10 years on
average from disease onset.
About VYNDAQEL® (tafamidis 61 mg) and VYNDAQEL®
(tafamidis meglumine 20 mg) 1,6
VYNDAQEL (tafamidis 61 mg) and VYNDAQEL (tafamidis meglumine 20
mg) are oral transthyretin stabilizers that selectively bind to
transthyretin, stabilizing the tetramer of the transthyretin
transport protein and slowing the formation of amyloid that causes
ATTR-CM.
The tafamidis 61 mg capsule corresponds to an 80 mg tafamidis
meglumine dose (4x 20mg capsules) and was developed for patient
convenience to enable a single capsule for daily administration.
VYNDAQEL 61 mg and VYNDAQEL 20 mg are not substitutable on a per
milligram basis.
Tafamidis was granted Orphan Drug Designation for ATTR-CM in
both the EU and US in 2012 and in Japan in 2018. Tafamidis was
approved in Japan under SAKIGAKE designation for the treatment of
ATTR-CM in March 2019, in the United States in May 2019, and in the
United Arab Emirates in November 2019.
VYNDAQEL (20 mg) was first approved in 2011 in the EU for the
treatment of transthyretin amyloid polyneuropathy (ATTR-PN), in
adult patients with stage 1 symptomatic polyneuropathy to delay
peripheral neurologic impairment. Currently, it is approved for
ATTR-PN in 45 countries, including Japan, countries in Europe,
Brazil, Mexico, Argentina, Israel, Russia, and South Korea.
VYNDAQEL has not received marketing authorisation for patients
with ATTR-CM in the EU.
VYNDAQEL® (tafamidis meglumine) and VYNDAMAX™
(tafamidis) From the U.S. Important Safety Information
Adverse Reactions
In studies in patients with ATTR-CM the frequency of adverse
events in patients treated with VYNDAQEL was similar to
placebo.
Specific Populations
Pregnancy: Based on findings from animal studies,
VYNDAQEL and VYNDAMAX may cause fetal harm when administered to a
pregnant woman.
Lactation: There are no available data on the presence of
tafamidis in human milk, the effect on the breastfed infant, or the
effect on milk production. Tafamidis is present in rat milk. When a
drug is present in animal milk, it is likely the drug will be
present in human milk. Breastfeeding is not recommended during
treatment with VYNDAQEL and VYNDAMAX.
The full prescribing information for VYNDAQEL and VYNDAMAX can
be found here
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DISCLOSURE NOTICE: The information contained in this release is
as of December 13, 2019. Pfizer assumes no obligation to update
forward-looking statements contained in this release as the result
of new information or future events or developments.
This release contains forward-looking information about a
potential indication in the EU for Vyndaqel for the treatment of
wild-type or hereditary transthyretin amyloidosis in adult patients
with cardiomyopathy (the “Potential Indication”) and Pfizer’s rare
disease portfolio, including their potential benefits, that
involves substantial risks and uncertainties that could cause
actual results to differ materially from those expressed or implied
by such statements. Risks and uncertainties include, among other
things, the uncertainties inherent in research and development,
including the ability to meet anticipated clinical endpoints,
commencement and/or completion dates for our clinical trials,
regulatory submission dates, regulatory approval dates and/or
launch dates, as well as the possibility of unfavorable new
clinical data and further analyses of existing clinical data; the
risk that clinical trial data are subject to differing
interpretations and assessments by regulatory authorities; whether
regulatory authorities will be satisfied with the design of and
results from our clinical studies; whether and when any new or
supplemental drug applications may be filed in any other
jurisdictions for tafamidis for the Potential Indication; whether
and when the EMA may approve the pending application for tafamidis
for the Potential Indication and whether and when regulatory
authorities in any such other jurisdictions where applications for
tafamidis may be pending or filed for the Potential Indication or
any other potential indications for tafamidis may approve any such
applications, which will depend on myriad factors, including making
a determination as to whether the product’s benefits outweigh its
known risks and determination of the product’s efficacy and, if
approved, whether tafamidis will be commercially successful;
decisions by regulatory authorities impacting labeling,
manufacturing processes, safety and/or other matters that could
affect the availability or commercial potential of tafamidis,
including for the Potential Indication; and competitive
developments.
A further description of risks and uncertainties can be found in
Pfizer’s Annual Report on Form 10-K for the fiscal year ended
December 31, 2018 and in its subsequent reports on Form 10-Q,
including in the sections thereof captioned “Risk Factors” and
“Forward-Looking Information and Factors That May Affect Future
Results”, as well as in its subsequent reports on Form 8-K, all of
which are filed with the U.S. Securities and Exchange Commission
and available at www.sec.gov and www.pfizer.com.
References
1 Rapezzi C, Lorenzini M, Longhi S, et al. Cardiac amyloidosis:
the great pretender. Heart Fail Rev. 2015;20(2):117-124.
2 Ando Y, Coelho T, Berk JL, et al. Guideline of
transthyretin-related hereditary amyloidosis for clinicians. Orph J
of Rare Diseases. 2013;8:31.
3 Rapezzi C, Quarta CC, Riva L, et al. Transthyretin related
amyloidoses and the heart: a clinical overview. Nat Rev Cardiol.
2010;7:398-408.
4 Castano A, Drachman BM, Judge J, et al. Natural history and
therapy of TTR-cardiac amyloidosis: emerging disease-modifying
therapies from organ transplantation to stabilizer and silencer
drugs. Heart Fail Rev. 2015; 20(2): 163–178.
doi:10.1007/s10741-014-9462-7.
5 Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis
treatment for patients with transthyretin amyloid cardiomyopathy. N
Engl J Med. 2018;379(11):1007-1016.
6 Data on file. Pfizer Inc. New York, NY.
7 Connors LH, Sam F, Skinner M, et al. Heart failure due to
age-related cardiac amyloid disease associated with wild-type
transthyretin: a prospective, observational cohort study.
Circulation. 2016;133(3):282-290.
8 Swiecicki PL, Zhen DB, Mauermann ML, et al. Hereditary ATTR
amyloidosis: a single-institution experience with 266 patients.
Amyloid. 2015;22(2):123-131.
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